Promising New Therapy For MS Patients

An article posted early online from the Archive of Neurology reports on a
promising new treatment for people with multiple sclerosis (MS). Researchers
have found that high doses of the drug cyclophosphamide, an immunosuppressant,
have reduced MS activity and disability in people with an aggressive form of the
disease.

Multiple sclerosis (MS) is an autoimmune condition in which the
immune system attacks the central nervous system, leading to the degeneration of
the coating that protects nerve cells (called the myelin sheath). The
inflammatory disease impairs physical and cognitive function by impeding the
nervous system's ability to send electrical signals. Each of the four identified
MS subtypes has a distinct autoimmune process, and this has made it difficult to
find optimal therapies that can intervene with the immune system to treat
MS.

With mixed results, the drug cyclophosphamide has been used to treat
MS because it affects the functions of the immune system's T and B cells. The
therapy is usually combined with bone marrow transplantation. To further
investigate how high doses of the drug (without bone marrow transplantation)
affect patients with aggressive relapsing-remitting MS, Chitra Krishnan, M.H.S.
(Bloomberg School of Public Health, Johns Hopkins University, Baltimore) and
colleagues conducted a two-year open-label trial with six men and three women
who averaged 35 years of age. In relapsing-remitting MS (the most common form),
patients experience both periods of symptoms and periods of symptom-free
remission. The treatment consisted of 50 milligrams per kilogram per day of
cyclophosphamide, injected intravenously for four consecutive
days.

During the 23 months (average) of follow-up, none of the patients
died or had unexpected or serious adverse events. The researchers found that on
tests measuring physical and mental function, the patients recorded an 87%
improvement. They also saw a 39.4% decrease in disability. Measuring the average
number of MS-related brain lesions using magnetic resonance imaging (MRI), the
researchers found a significant decrease from 6.5 to 1.2
lesions.

"High-dose cyclophosphamide treatment of patients with
aggressive MS was safe and well tolerated and did not lead to excess morbidity
or accelerated brain atrophy," highlight the authors. "Moreover, high-dose
cyclophosphamide induced a functional improvement in most of the patients we
studied. In many of those patients, the functional improvement was sustained
through the length of the study (up to 24 months) despite the absence of any
immunomodulatory therapies beyond the initial high-dose cyclophosphamide
treatment."

They conclude that, "This immunoablative regimen [an
immune-related therapy involving the destruction of a cell population] of
cyclophosphamide for patients with aggressive MS is worthy of further study and
may be an alternative to bone marrow transplantation."